PB1805: INSIGHTS FROM ORAL AZACITIDINE EARLY ACCESS PROGRAM IN FRANCE: PATIENT POPULATION WITH ACUTE MYELOID LEUKEMIA RECEIVING ORAL-AZA FOR MAINTENANCE OF COMPLETE REMISSION AFTER INDUCTION CHEMOTHERAPY
Authors: E. Raffoux, S. De Botton, L. Ades, M. Carré, T. Cluzeau, C. Saillard, M. Heiblig, F. Beckerich, L. Gastaud, A. Dupuis, U. Richez, P.-Y. Dumas
Journal: HemaSphere (2022)

Background: While many older patients with acute myeloid leukemia (AML) reach complete remission (CR) with intensive chemotherapy (IC), ~80% will ultimately relapse. Treatment options in relapse settings are limited and outcome is usually poor. The randomized, placebo-controlled, phase 3 QUAZAR AML-001 trial evaluated oral azacitidine (Oral-AZA; formerly CC-486), a hypomethylating agent, in patients with AML in first remission (CR1) after IC, who were not eligible for hematopoietic stem cell transplantation (HSCT). Unprecedentedly in AML, maintenance treatment with Oral-AZA was associated with significantly longer overall survival and relapse-free survival compared with placebo. Therefore, Oral-AZA received a marketing authorization in the EU as maintenance therapy in adult patients with AML who achieved CR or CR with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, HSCT. Also, French health Authority enabled an early access to Oral-AZA in this indication via a Temporary Authorization for Use (ATU) that started in June 2021. Aims: The aim of the study was to describe the characteristics of patients with newly diagnosed AML who received Oral-AZA for maintenance of CR/CRi after IC and consolidation treatment, who were not eligible for HSCT and were enrolled into ATU in France. Methods: ATU is a French early access allowing administration of the drug under a Therapeutic Use Protocol approved by health authorities. Data presented in this abstract were collected on a declaratory basis and were not monitored. A total of 52 patients were included in 32 centers across France and treated with Oral-AZA during the period from 09-Jun-2021 to 14-Sep-2021. Oral-AZA monotherapy was administered at 300mg once daily for 14 days per 28-days cycle. Results: Overall, 52 treated patients were included with a median age of 67.5 years [range: 35-85]. Regarding Performance Status, 22/52 (42.3%) had an ECOG 0, 25/52 (48.1%) 1 and 5/52 (9.6%) ≥2. The majority (43/52 [82.7%]) had a diagnosis of de novo AML, and the genetic risk (according to ELN2017 criteria) was favorable for 13/52 (25.0%), intermediate for 22/52 (42.3%) and adverse for 17/52 (32.7%) of the patients. Most of the patients had received 1 cycle of IC induction (45/52 [86.5%]). The induction regimen was 3 + 7 for 42/52 (80.8%) patients, among those, 3 (5.8%) received midostaurin along with the 3 + 7. Also 7/52 (13.5%) received CPX-351 as induction regimen. The median number of consolidation cycles was 2 [range: 0-6]; 8/52 (15.4%) patients did not receive any consolidation and 6/52 (11.5%) had >3 cycles of consolidation. The majority of patients (27/52 [61.4%]) received IDAC consolidation. The median time between AML diagnosis and Oral-AZA ATU request approval was 6.8 months [range: 1-98]. Because of the limited time frame of the ATU, follow-up is available for 14/52 patients, and the median follow-up is 21.8 days [range: 1-35]. During the follow-up period, one patient had a temporary interruption of Oral-AZA treatment due to grade 4 neutropenia. There was no reported permanent discontinuation of treatment related to an adverse event. Summary/Conclusion: The characteristics of the patients included in the Oral-AZA Early Access Program in France are in line with European label. These real-life results bring new insights in this population of patients who are not eligible to HSCT with high unmet need to prolong CR1. No unexpected safety signal was registered. Updated results will be presented at the meeting.